- Trials with a EudraCT protocol (8)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
8 result(s) found for: Splice site mutations.
Displaying page 1 of 1.
EudraCT Number: 2020-002337-15 | Sponsor Protocol Number: 99625945 | Start Date*: 2020-09-16 |
Sponsor Name:Oslo University Hospital | ||
Full Title: TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS (THE GENTELBULL STUDY) | ||
Medical condition: Epidermolysis bullosa caused by nonsense mutations or splice site mutations | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |
Trial protocol: NO (Completed) | ||
Trial results: (No results available) |
EudraCT Number: 2019-003159-12 | Sponsor Protocol Number: J4B-MC-OKAA | Start Date*: 2021-12-24 | |||||||||||
Sponsor Name:Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company | |||||||||||||
Full Title: A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM) | |||||||||||||
Medical condition: Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: BE (Trial now transitioned) ES (Temporarily Halted) FR (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2016-000925-38 | Sponsor Protocol Number: UCL13/0076 | Start Date*: 2017-01-04 | ||||||||||||||||
Sponsor Name:University College London (UCL) | ||||||||||||||||||
Full Title: GO-8: Gene therapy for haemophilia A using a novel serotype 8 capsid pseudotyped adeno-associated viral vector encoding Factor VIII-V3 | ||||||||||||||||||
Medical condition: Severe Haemophilia A | ||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male | |||||||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-006574-23 | Sponsor Protocol Number: AR101-PREVEnt | Start Date*: 2022-08-30 | |||||||||||
Sponsor Name:Aytu BioPharma, Inc. | |||||||||||||
Full Title: A multicenter, randomized, double-blind, placebo-controlled study of enzastaurin for the prevention of arterial events in patients with vascular Ehlers-Danlos Syndrome (vEDS) confirmed with COL3A1 ... | |||||||||||||
Medical condition: vascular Ehlers-Danlos Syndrome (vEDS) confirmed with COL3A1 mutations | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: SE (Ongoing) BE (Prematurely Ended) FR (Ongoing) NL (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2022-000691-19 | Sponsor Protocol Number: BN43881 | Start Date*: 2022-10-26 | ||||||||||||||||
Sponsor Name:F. Hoffmann-La Roche Ltd | ||||||||||||||||||
Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy | ||||||||||||||||||
Medical condition: Duchenne Muscular Dystrophy | ||||||||||||||||||
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Population Age: Infants and toddlers, Children, Under 18 | Gender: Male | |||||||||||||||||
Trial protocol: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2022-003407-15 | Sponsor Protocol Number: SRP-9001-104 | Start Date*: 2023-08-08 |
Sponsor Name:Sarepta Therapeutics, Inc | ||
Full Title: An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in association with imlifidase in Subjects with Duchenne Muscular Dystrophy with pre-exis... | ||
Medical condition: Duchenne muscular dystrophy | ||
Disease: | ||
Population Age: Children, Under 18 | Gender: Male | |
Trial protocol: ES (Temporarily Halted) | ||
Trial results: (No results available) |
EudraCT Number: 2019-003374-91 | Sponsor Protocol Number: SRP-9001-301 | Start Date*: 2022-09-12 | ||||||||||||||||
Sponsor Name:Sarepta Therapeutics, Inc. | ||||||||||||||||||
Full Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBA... | ||||||||||||||||||
Medical condition: Duchenne Muscular Dystrophy | ||||||||||||||||||
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Population Age: Children, Under 18 | Gender: Male | |||||||||||||||||
Trial protocol: FR (Completed) BE (Completed) ES (Completed) DE (Completed) IT (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2014-002714-23 | Sponsor Protocol Number: LLC1114 | Start Date*: 2015-01-07 | |||||||||||
Sponsor Name:Fondazione G.I.M.EM.A. Onlus Gruppo Italiano Malattie Ematologiche dell'Adulto | |||||||||||||
Full Title: A phase 2 multicenter study to assess the activity and the safety of front-line Ibrutinib plus Rituximab (IR) in unfit patients with Chronic Lymphocytic Leukemia (CLL). | |||||||||||||
Medical condition: Chronic Lymphocytic Leukemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) | |||||||||||||
Trial results: (No results available) |
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